News

FDA Grants Rare Pediatric Disease Designation to ABO-202 for Infantile Batten Disease

The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202 program being developed for Batten disease caused by mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis. ABO-202 is an adeno-associated virus (AAV)-based gene therapy (AAV-CLN1) where weakened…

BioMarin Launches ‘RARE Scholars’ Scholarship Program for Students with Batten, Other Rare Diseases

BioMarin recently announced the launch of RARE Scholars, an annual scholarship program for students with certain rare diseases, including Batten disease, who demonstrate strong academic performance, leadership skills, and participation in school and community activities. The program is designed to assist high school seniors, graduates, current postsecondary undergraduates, or…